I consider watching our daughter struggle through the symptoms of Lambert-Eaton myasthenic syndrome (LEMS) one of the most difficult aspects of my life. As I look back on the last two and a half years since Grace began showing symptoms, I realize how I haphazardly stumbled through the five stages of...
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Busan, South Korea – Head and neck squamous cell carcinoma (HNSCC) is a type of cancer that affects the mucous membranes of the mouth, nose, and throat. HNSCC is typically associated with tobacco exposure, alcohol abuse, and viral infections. The links between human papillomavirus (HPV) infection status and the molecular...
ARLINGTON, Va. — The ALS Association proudly announces the largest single gift in its history from the estate of the late philanthropist Hugh Hoffman. The $58 million gift will profoundly impact the lives of people living with ALS and their loved ones, advancing the ALS Association’s goal of making amyotrophic...
SHANGHAI and CLINTON, N.J. — HuidaGene Therapeutics today announced the U.S. FDA granted Rare Pediatric Drug Designation (RPDD) to HG302, a novel CRISPR-Cas12 DNA-editing therapy, to treat Duchenne muscular dystrophy (DMD), affecting 1 in ~5,000 newborn boys. Unlike the in-vivo use of Cas9 system with limited genome-editing efficiency, high occurrence...
LONDON and NEW YORK – Huma, Europe’s fastest-growing health technology company [1], is partnering with the non-profit patient advocacy group RARE-X, which runs a collaborative patient-powered data collection platform for global data sharing and analysis to accelerate treatments for rare disease. RARE-X’s federated data platform will pair with Huma’s decentralized clinical trials platform, complete...
Human fibrinogen concentrate (HFC) has favorable pharmacokinetics for adult, adolescent, and pediatric patients with congenital fibrinogen deficiency (CFD), according to the results of open-label studies presented at the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) meeting. The prospective studies FORMA-01, FORMA-02, and FORMA-04 looked at efficacy, safety, and dosing of...
Human fibrinogen concentrate (HFC) has favorable pharmacokinetics for adult, adolescent, and pediatric patients with congenital fibrinogen deficiency (CFD), according to the results of open-label studies presented at the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) meeting. The prospective studies FORMA-01, FORMA-02, and FORMA-04 looked at efficacy, safety, and dosing of...
ROCKVILLE, Md. — Human Genome Sciences, Inc. (Nasdaq: HGSI) today announced the presentation of results from a long-term Phase 2 continuation trial showing that BENLYSTA(TM) (belimumab, formerly LymphoStat-B(R)) was associated with sustained improvement in disease activity across multiple clinical measures, decreased frequency of disease flares, and was generally well tolerated...
Houston, TX. – Researchers at Baylor College of Medicine and collaborating institutions working with human intestinal organoids, also called mini guts, have shed new light on the potential causes of Cronkhite-Canada syndrome, a rare condition characterized by abundant non-cancerous growths or polyps in the intestine and other symptoms such as...
BURLINGAME, Calif. – Humanigen, Inc. (Nasdaq:HGEN), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab™, today announced positive results from the Phase 1 safety and bioimaging trial of its second Humaneered® antibody, ifabotuzumab, in patients with glioblastoma multiforme...